Project Title: Rare Diseases Clinical Research Scholar's Program: PROJECT SUMMARY Rare diseases (RD) are unique as a medical field, comprising a large number of disorders with a small number of patients in each. They primarily affect children and most have a genetic basis. The methods employed in rare disease research (RDR) require models and analytical tools that are different from more common conditions. This proposal seeks to develop a blended learning curriculum employing specific tools and methods unique to rare diseases. It will continue to be work closely with the NIH funded Rare Diseases Clinical Research Network (RDCRN), a collaborative of 240 sites at universities and academic medical centers. It is expected that this program will continue to attract and retain new investigators in the field of rare disease through network development, research success, and exposure to patient and family groups. The Rare Disease Scholar's Program is a curriculum provided over an academic year that has 6 components: 1.) an initial two day face-to-face RDR methods and policy workshop that will bring together the trainees, mentors, patient advocacy groups, federal agencies and biotech/pharma. 2.) a series of semi-monthly interactive, web-based seminars on methodologies in RDR including study design, statistics, bioethics, therapeutics, policy and approaches to interacting with other key contributors to the field, including patient advocacy groups, federal agencies and biotech/pharma. 3.) a semi-monthly ?chalk board? videoconference focusing on individual trainee RDR projects with an emphasis on practical design and analytical tools. 4) a web portal with course content, opportunities for interaction among trainees and between trainees and teachers/mentors, and other interactive features. 5) a ?capstone? research project presentation by each of the trainees at the semi-annual RDCRN meeting that will bring together the trainees, patient advocacy groups, federal agencies and biotech/pharma. 6.) Utilizing the CTSA tracking and evaluation system and a pre- and posttest to quantify understanding of the topics involved in RDR prior to and following the curriculum. These tools will also be used to monitor the quality of the program and instruction. We will track the program's outcome by following the trainees in terms of academic career choice, retention in the field of RDR, and other measures of success. Twenty to twenty five trainees will continue to matriculate each year. The program involves 80 hours of direct teaching and at least 40 hours of prep work (homework) as well as the work required to draft and carry out a research study protocol, and write up the results. Trainees completing the program will receive a certificate in RDR from the RDCRN.